@article{314d4624706f44759cd3c72353bbc89a,
title = "Evidence- and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria",
abstract = "Purpose: Phenylketonuria (PKU) is a rare metabolic disorder that requires life-long management to reduce phenylalanine (Phe) concentrations within the recommended range. The availability of pegvaliase (PALYNZIQ{\texttrademark}, an enzyme that can metabolize Phe) as a new therapy necessitates the provision of guidance for its use. Methods: A Steering Committee comprising 17 health-care professionals with experience in using pegvaliase through the clinical development program drafted guidance statements during a series of face-to-face meetings. A modified Delphi methodology was used to demonstrate consensus among a wider group of health-care professionals with experience in using pegvaliase. Results: Guidance statements were developed for four categories: (1) treatment goals and considerations prior to initiating therapy, (2) dosing considerations, (3) considerations for dietary management, and (4) best approaches to optimize medical management. A total of 34 guidance statements were included in the modified Delphi voting and consensus was reached on all after two rounds of voting. Conclusion: Here we describe evidence- and consensus-based recommendations for the use of pegvaliase in adults with PKU. The manuscript was evaluated against the Appraisal of Guidelines for Research and Evaluation (AGREE II) instrument and is intended for use by health-care professionals who will prescribe pegvaliase and those who will treat patients receiving pegvaliase.",
keywords = "PALYNZIQ, pegvaliase, anaphylaxis management, dietary management, hypophenylalaninemia",
author = "Nicola Longo and David Dimmock and Harvey Levy and Krista Viau and Heather Bausell and Bilder, {Deborah A.} and Barbara Burton and Christel Gross and Hope Northrup and Fran Rohr and Stephanie Sacharow and Amarilis Sanchez-Valle and Mary Stuy and Janet Thomas and Jerry Vockley and Roberto Zori and Harding, {Cary O.}",
note = "Funding Information: N.L. has participated as an investigator in clinical trials sponsored by Aeglea, BioMarin Pharmaceutical Inc., Genzyme, Horizon Pharma, Protalix, Retrophin, Shire, Stealth Therapeutics, and Ultragenyx. He is on the Steering Committee for the pegvaliase clinical trial program. He has received grants from Lumos Pharma and consultancy fees from Aeglea, BioMarin Pharmaceutical Inc., Censa Pharmaceuticals, Dimension Therapeutics, Genzyme, Hemoshear, Horizon Pharma, Lumos Pharma, Moderna, Mitobridge, Pfizer, Retrophin, and Stealth Therapeutics. He has received travel fees from BioMarin Pharmaceutical Inc., Cello Health Sciences, Lumos Pharma, and SigmaTau/ AlphaSigma. D.D. has (in the past 5 years) participated as an investigator in clinical trials sponsored by BioMarin Pharmaceutical Inc., Genzyme/Sanofi, and Amicus Therapeutics. He has received research support and consultancy fees from BioMarin Pharmaceutical Inc. and Demeter Therapeutics. He is a member of the Scientific and Clinical Advisory Board at Audentes Therapeutics Inc. and has served as a consultant for Ichorion Therapeutics and Complete Genomics. H.L. is involved in clinical trials sponsored by BioMarin Pharmaceutical Inc., has acted as a consultant, and has participated in advisory boards for BioMarin Pharmaceutical Inc. K.V. has been involved in clinical trials sponsored by BioMarin Pharmaceutical Inc., Horizon Pharma, and Ultragenyx. She has received consultancy fees from BioMarin Pharmaceutical Inc. and Horizon Pharma and has participated in advisory boards held by BioMarin Pharmaceutical Inc. H.B. has been involved in clinical trials sponsored by BioMarin Pharmaceutical Inc. and Ultragenyx. She has received consultancy fees from BioMarin Pharmaceutical Inc., Horizon Pharma, Cambrooke Therapeutics, and Nutricia. D.A.B. has been involved in a phase 3 study sponsored by BioMarin Pharmaceutical Inc., has participated in advisory boards held by BioMarin Pharmaceutical Inc. and Audentes Therapeutics Inc., and has received consultancy fees from BioMarin Pharmaceutical Inc. B.B. has participated as an investigator for clinical trials sponsored by by Alexion, BioMarin Pharmaceutical Inc., Genzyme Sanofi, Shire, and Ultragenyx Pharmaceuticals Inc. She has participated in advisory boards for BioMarin Pharmaceutical Inc., Horizon Pharma, Nestle, and Viking Therapeutics, and has received research grants from Shire and consultancy fees from Alexion, Shire, BioMarin Pharmaceutical Inc., and RegenxBio. C.G. is involved in clinical trials sponsored by BioMarin Pharmaceutical Inc. H.N. has been involved in clinical trials sponsored by BioMarin Pharmaceutical Inc. and The University of Texas Health Science Center, Houston, and has participated in advisory boards held by and received consultancy fees from BioMarin Pharmaceutical Inc. F.R. has been involved in clinical trials sponsored by BioMarin Pharmaceutical Inc. and has received consultancy fees from BioMarin Pharmaceutical Inc. S.S. is an investor in clinical trials sponsored by BioMarin Pharmaceutical Inc. and has participated in advisory boards held by BioMarin Pharmaceutical Inc. A.S.‐V. has participated as an investigator for clinical trials sponsored by Alexion, BioMarin Pharmaceutical Inc., Genzyme, Horizon Pharma, and Ultragenyx Pharmaceuticals Inc. She has participated in advisory boards for BioMarin Pharmaceutical Inc. and Horizon Pharma. M.S. has participated as a principal investigator in clinical trials sponsored by BioMarin Pharmaceutical Inc. J.T. has been involved in clinical trials sponsored by BioMarin Pharmaceutical Inc., and has participated in advisory boards held by BioMarin Pharmaceutical Inc. She is a member of the Steering Committee for the pegvaliase clinical program. J.V. has participated as an investigator in clinical trials sponsored by BioMarin Pharmaceutical Inc. and has received research support from BioMarin Pharmaceutical Inc. He was a speaker at the BC Childrens{\textquoteright} and Womens{\textquoteright} Hospital in Vancouver on behalf of the Canadian PKU Alliance; this event was sponsored by BioMarin Pharmaceutical Inc., Cambrooke Therapeutics, Mead Johnson, Nutricia, and Vitaflo. R.Z. has participated as an investigator in clinical trials sponsored by BioMarin Pharmaceutical Inc. and Hyperion Therapeutics Inc. and is on the Steering Committee for the pegvaliase clinical program. C.O.H. has participated as an investigator for clinical trials sponsored by BioMarin Pharmaceutical Inc. and has received consultancy fees services and research grants from BioMarin Pharmaceutical Inc., Cydan Development Inc., Dimension Therapeutics, Horizon Pharma, Pfizer, Rubius Therapeutics, StrideBio, and Synlogic. Publisher Copyright: {\textcopyright} 2018, The Author(s).",
year = "2019",
month = aug,
day = "1",
doi = "10.1038/s41436-018-0403-z",
language = "English (US)",
volume = "21",
pages = "1851--1867",
journal = "Genetics in Medicine",
issn = "1098-3600",
publisher = "Lippincott Williams and Wilkins",
number = "8",
}